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Why we should care about ultra-rare disease
5 references·0 citing·22 second hop
Why we should care about ultra-rare disease (2016)Why we should care about ultra-rare disea…The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review (2015)The quality of economic evalu…Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders (2014)Incremental cost per quality-…Orphan drug development is not taking off (2009)Orphan drug development is no…A journey of hope: lessons learned from studies on rare diseases and orphan drugs (2006)A journey of hope: lessons le…Drugs for exceptionally rare diseases: do they deserve special status for funding? (2005)Drugs for exceptionally rare …National Institute for Clinical Excellence and its value judgments (2004)Criteria list for assessment of methodological quality of economic evaluations: Consensus on Health Economic Criteria (2005)Cochrane Handbook for Systematic Reviews of Interventions Version 5.0.1. The Cochrane Collaboration (2008)Affordable orphan drugs: a role for not‐for‐profit organizations (2017)Economic Modeling Considerations for Rare Diseases (2018)How to Value Orphan Drugs? A Review of European Value Assessment Frameworks (2021)Orphan drug development is progressing too slowly (2006)Rare diseases and orphan drugs (2006)Why rare diseases are an important medical and social issue (2008)An Empirical Review of Major Legislation Affecting Drug Development: Past Experiences, Effects, and Unintended Consequences (2011)Rare lung disease and orphan drug development (2013)A theory of justice original edition (1971)Cost-Effectiveness in Health and Medicine (1996)Methods for the Economic Evaluation of Health Care Programmes (2005)Health technology assessment (HTA) and economic evaluation: efficiency or fairness first (2018)Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group) (2021)Setting Health Care Priorities in Oregon (1991)Intergenerational Equity: An Exploration of the ‘Fair Innings’ Argument (1997)QALY maximisation and people's preferences: a methodological review of the literature (2004)Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients’ health needs? (2017)Challenges in Research and Health Technology Assessment of Rare Disease Technologies: Report of the ISPOR Rare Disease Special Interest Group (2018)Incentives for orphan drug research and development in the United States (2008)
this work references cited by second hop

References

  • 2005Drugs for exceptionally rare diseases: do they deserve special status for funding?220 cited
  • 2006A journey of hope: lessons learned from studies on rare diseases and orphan drugs185 cited
  • 2015The quality of economic evaluations of ultra-orphan drugs in Europe – a systematic review75 cited
  • 2014Incremental cost per quality-adjusted life year gained? The need for alternative methods to evaluate medical interventions for ultra-rare disorders73 cited
  • 2009Orphan drug development is not taking off63 cited

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